New Drug Approvals: Which Drugs Made the Mark in 2018?

By Patricia Van Arnum - DCAT Editorial Director

January 16, 2019

Last year (2018) was a record-setting year for approvals of new molecular entities (NMEs) by the FDA with 59 NMEs approved. DCAT Value Chain Insights examines the 2018 NME approvals, the product mix between small molecules and biologics, and which pharma companies led the pack.

Inside new drug approvals

In 2018, the US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) approved a record-high 59 new molecular entities (NMEs), surpassing the previous record of 53 NME approvals in 1996 and the 46 NMEs approved in 2017. The 59 NME approvals in 2018 was nearly double the 10-year (2009–2017) average of 33 NME approvals by FDA’s CDER. The record-number of NME approvals continues an upward trajectory for NME approvals (with the exception of 2016 when 22 NMEs were approved) over the last several years. FDA’s CDER approved 41 NMEs in 2014, 45 NMEs in 2015, 46 NMEs in 2017, and the 59 NMEs in 2018.

In addition, continuing a recent trend, orphan drugs accounted for a large percentage of NME approvals. More than half (58%), or 34, of the NME approvals in 2018 were for orphan drugs, defined as drugs that treat 200,000 or fewer people in the US. In 2017, 39%, or 18 of the 46 NMEs approved by the FDA’s CDER were orphan drugs, which continued a recent trend of approximately 40% of NME approvals being orphan drugs. In 2016, 9 of the 22 NMEs approved by the FDA were orphan drugs. In 2015, 47%, or 21 of the 45 NMEs approved by the FDA’s CDER were orphan drugs, and in 2014, 41%, or 17 of the 41 NMEs approved were orphan drugs.

Inside NME approvals in 2018

Table I at the end of article outlines the NMEs approved in 2018 which was second only to the 53 NMEs approved in 1996. Small molecules dominated with 42 small molecule NME approvals (72% of NME approvals) and 17 biologic-based NME approvals (28% of NME approvals), which includes one antibody drug conjugate (ADC). This product mix in 2018 is consistent with NME approvals in 2017. In 2017, small-molecule NME approvals accounted for 76% of new drug approvals (see Table II at end of article) and biologics 24%.

The large pharmaceutical companies with biologic-based drug approvals in 2018 include: Amgen’s Aimovig (erenumab-aooe), a drug to prevent migraines in adults; AstraZeneca’s Lumoxiti (moxetumomab pasudotox-tdfk), an ADC for treating hairy cell leukemia; Eli Lilly and Company’s Emgality (galcanezumab-gnlm) for the preventive treatment of migraine in adults; Merck & Co.’s and Sun Pharmaceutical's Ilumya (tildrakizumab) for treating moderate-to-severe plaque psoriasis; and Teva Pharmaceutical Industries’ Ajovy (fremanezumab-vfrm) for the preventive treatment of migraine in adults. Table I at the end of the article summarizes approvals for biologics [denoted by BLA for biologics license applications (BLA) and small-molecules (denoted by new drug application (NDA)] in 2018.

Big Pharma and NME approvals

Table I: Approvals of New Molecular Entities, New Drug Applications (NDAs) and Original Biologics License Applications (BLAs) by the US Food and Drug Administration’s Center in 2018.

Company

Brand name (active ingredient); application type

Indication

AbbVie

Orilissa (elagolix sodium); NDA

Moderate-to-severe pain associated with endometriosis

Advanced Accelerator Applications (Novartis company)

Lutathera (lutetium Lu 177 dotatate); NDA

Gastroenteropancreatic neuroendocrine tumors, a type of cancer that affects the pancreas or gastrointestinal tract

Achaogen

Zemdri (plazomicin); NDA

Adults with complicated urinary tract infections

Agios Pharmaceuticals

Tibsovo (ivosidenib); NDA

Relapsed or refractory acute myeloid leukemia

AkaRx

Doptelet (avatrombopag); NDA

Low blood platelet count (thrombocytopenia) in adults with chronic liver disease who are scheduled to undergo a medical or dental procedure

Alexion Pharmaceuticalstd>

Ultomiris (ravulizumab); BLA

Paroxysmal nocturnal hemoglobinuria

Allergan

Seysara (sarecycline); NDA

Inflammatory lesions of non-nodular moderate to severe acne vulgaris in patients 9 years of age and older

To treat adults and children ages 1 year and older with x-linked hypophosphatemia, a rare, inherited form of rickets

US WorldMeds

Lucemyra (lofexidine); NDA

Non-opioid treatment for management of opioid withdrawal symptoms in adults

Vertex Pharmaceuticals

Symdeko (tezacaftor and ivacaftor); NDA

Cystic fibrosis in patients age 12 years and older

Novartis acquired Advanced Accelerator Applications in January 2018.

In August 2015, Amgen entered into a global collaboration with Novartis to jointly develop and commercialize treatments in the fields of migraine and Alzheimer's disease. The collaboration focuses on investigational Amgen drugs in the migraine field, including Aimovig. In April 2017, the collaboration was expanded to include co-commercialization of Aimovig in the US. For the migraine program, Amgen retains exclusive rights in Japan, and Novartis has exclusive rights in Europe, Canada, and rest of the world.

Array BioPharma has exclusive rights to Braftovi (encorafenib) and Mektovi (binimetinib) in the US and Canada. Array has granted Ono Pharmaceutical exclusive rights to commercialize both products in Japan and South Korea and Pierre Fabre exclusive rights to commercialize both products in all other countries, including in Europe, Asia, and Latin America.

Eli Lilly and Company agreed to acquire Loxo Oncology in 2018.

Shire was acquired by Takeda in January 2018.

Sun Pharmaceutical Industries Ltd.'s wholly owned subsidiary licensed worldwide rights to Ilumya (tildrakizumab) from a subsidiary of Merck & Co., Inc. in 2014. Funded by a Sun Pharma subsidiary, Merck & Co., Inc. was responsible for the completion of Phase-III trials and submission of a biologics license application to the US Food and Drug Administration (FDA) as well as manufacturing finished goods to support Sun Pharma's initial product launch. Sun Pharma is responsible for all post-approval regulatory activities, including subsequent submissions, pharmacovigilance, post approval studies, manufacturing, and commercialization of the approved product. Sun Pharma is also responsible for all regulatory, pharmacovigilance, post approval studies, manufacturing, and commercialization of approved products for all non-U.S. markets. Merck & Co is eligible to receive milestone payments and royalties on sales of Ilumya.

Source: US Food and Drug Administration's Center for Drug Evaluation and Research and company information.

Table II: Small Molecule and Biologics New Molecular Entities Approved by the US Food and Drug Administration’s Center for Drug Evaluation and Research, 2010 to 2017.

Year

Number of New Molecular Entities (NMEs) Approved

Percentage relative to total NME approvals and number of small molecules and biologics approved as NMEs

n 2015, 32 small-molecule drugs were approved as NDAs and one insulin analog, Novo Nordisk's Tresiba (insulin degludec injection), a long-acting basal human insulin analog produced by a process that includes expression of recombinant DNA in Saccharomyces cerevisiae followed by chemical modification was approved as a NDA, not as a biologics license application.